ICER Weekly View 02-09-18

From the desk of Mitchell Stein


Good morning. The government is back open. What? It was open when you went to bed and you don’t know what I’m talking about? While you were sleeping, the government shut down and then opened back up. No biggie. What is a biggie is the budget deal that came along with the latest funding measure. It has many health funding components, including an earlier closing of the Medicare Part D donut hole. We’ll look at that and a lot more this morning. Here’s this week’s full rundown:

ICER in the news – Is Kymriah overpriced? Differing views

Inside the Beltway – Budget deal, Creates Act, drug price survey, states and Canadian imports, and review of 2017 state actions on drug prices

Industry Trends – Express Scripts Trend Report, drug coupon changes, generic approvals down, migraines, CRISPR, PTSD drug fails trial, and genomic blockchain cryptocurrency start-up

Company/Drug Specific –Bristol-Myers, Sanofi, and Gilead

Weekly View In-depth – Right to Try legislation

Now, onto the news.

ICER In The News

David Mitchell of Patients for Affordable Drugs believes Kymriah is overpriced and co-authored a Health Affairs blog making that point. Matthew Herper of Forbes reported on the piece mentioning ICER’s draft report, the draft conclusions of which found that the drug was, in fact, cost-effective at the stated price of $475,000. In addition to the story below, there were two other pieces discussing Kymriah, David Mitchell’s blog, and ICER’s work. The first appeared in The Philadelphia Inquirer and then without a paywall on MedicalXpress: Breakthrough cancer therapy raises tough questions about drug costs, value. The second appeared on FiercePharma: Novartis could cut its Kymriah price to $160,000 and keep its profit margins: study.

Patient Advocate Says Novartis’ $475,000 Breakthrough Should Cost Just $160,000

David Mitchell, 67, says he’s sure that Novartis’ Kymriah is a breakthrough medicine, and that he will need a similar medicine to treat his own blood cancer. He’s sure of something else, too: Novartis is charging too much.

Pharmaceutical News

Inside the Beltway/State Regulatory Developments

As noted above, overnight the Senate and House passed a mammoth budget deal along with the latest continuing resolution (CR) to fund the government. This should be the last CR as the budget deal outlines funding levels for the next two years. For Pharma World, the most significant piece of the deal is closing of the Medicare Part D donut hole a year earlier and changing the manufacturers’ responsibility for the costs from 50% to 70%. According to reports, Pharma furious after being “blindsided” in budget deal. You can read more about the health care impacts of the deal here.

More details on the donut hole changes are below, and Avalere has looked at the impact: Impact of Coverage Gap Discount Changes in Budget Agreement.

Drugmakers expect multi-billion dollar hit under Capitol Hill budget deal

Despite the popularity of the Creates Act (Coalition of 44 groups calls for passage of drug pricing bill), it was left out of the budget deal.

Popular bill to fight drug prices left out of budget deal

Drug pricing advocates are decrying the budget deal announced Wednesday for leaving out a bipartisan drug pricing measure that they had pushed for. The measure would prevent branded drug companies from using delay tactics to prevent cheaper generic competitors from coming onto the market.

Lots of interesting numbers in this survey, with the main takeaway being the public still perceives prescription drug prices to be a problem that they overwhelmingly want the government to handle.

Poll: Majority of voters say lowering drug prices should be priority for Congress

More than 80 percent of voters think lowering drug prices should be a priority for Congress, according to a new poll released Monday. The poll, completed by GS Strategy Group, shows 85.5 percent of registered voters surveyed think lowering the cost of prescription drugs should be a “top priority” or an “important priority” for Congress.

Four more states move bills forward to allow importation of drugs from Canada.

Four More States Submit Bills to Import Prescription Drugs from Canada – NASHP

As states pursue a wide range of legislation to address rising drug costs, four more states have joined Utah and Vermont to introduce bills to import prescription drugs from Canada through a state-run, wholesale operation. This market-based approach to providing more affordable medicines from Canada, where prescription drugs cost on average 30 percent less than …

The states continue to be the place for activity on drug pricing measures. The Pew Charitable Trust runs down all of 2017’s state-based activity. 

States Use Various Approaches to Manage Drug Spending

Industry Trends

Express Scripts released their annual Drug Trend Report this week. Private plans’ (employer and individual) drug costs rose only 1.5% last year – the smallest increase since the pharmacy benefits company first began tracking spending in 1993.

Express Scripts plans see lower gains in prescription drug spending

Prescription drug prices may still be high, but Express Scripts says the commercial insurance plans it works with saw the lowest increase in drug spending last year in nearly a quarter of a century. For private employer and individual health plans, total drug spending rose 1.5 percent last year, according to the latest Express Scripts annual Drug Trend Report.

A change in the way drug coupons are handled (they will no longer count towards a patient’s deductible) will be a shock to patients and could cost some brand-name drugs significant sales.

The drug pricing issue that could erupt this year

Patients who use drug copay coupons may be surprised.

In January generic approvals failed to maintain the record pace achieved at the end of 2017, perhaps due to new impurities regulations.

Generic Drug Approvals Stall in January as Elemental Impurity Questions Linger

Posted 07 February 2018 | By Zachary Brennan The US Food and Drug Administration (FDA) approved generic drugs at record-high levels in 2017, though a new deadline related to elemental impurities has put a dent in that progress, at least preliminarily in January, according to the most recent generic drug activities report update.

NPR looks at new migraine treatments awaiting word of FDA approval. The treatments mentioned in this story are included in an upcoming ICER review.

Gone With A Shot? Hopeful New Signs Of Relief For Migraine Sufferers

Humans have suffered from migraines for millennia. Yet, despite decades of research, there isn’t a drug on the market today that prevents them by targeting the underlying cause. All of that could change in a few months when the FDA is expected to announce its decision about new therapies that have the potential to turn migraine treatment on its head.

Meanwhile, Teva’s migraine entrant hits a stumbling block.

In latest blow to Teva, CGRP migraine med could be delayed while rivals race towards the finish line

It’s starting to feel like Teva $TEVA can’t catch a break. After an incredibly rough series of events that led to massive layoffs, restructuring, and political upheaval, now Teva has quietly signaled to investors that the launch of its CGRP migraine med could be delayed. Teva’s stock has tumbled nearly

This CRISPR innovation could have far-reaching implications if the technique to address a single mutated allele is reproducible in other situations.

CRISPR trick in mice points the way to a possible treatment for blindness

t might seem that scientists have never met a chunk of DNA they couldn’t edit in mice or isolated cells using CRISPR – from mutations causing deafness to those for Duchenne muscular dystrophy. In fact, they are learning what every pencil- or Word-wielding editor knows: It’s much easier to improve something that’s in terrible shape than writing that’s near perfect.

A commonly used PTSD drug fails a large clinical trial.

A drug commonly used to treat PTSD symptoms fails in a clinical trial

housands of people with post-traumatic stress disorder have taken the drug prazosin to ease the nightmares and disturbances that stalk their sleep. Numerous studies have shown the drug to be effective at controlling those episodes. But a team of researchers from the Department of Veterans Affairs, seeking to collect more evidence, set out to study the sustained effectiveness of the treatment.

How many buzzwords can you put in your startup proposal? This one included genomics, blockchain, and cryptocurrency so they’re off to a good start. This announcement is getting attention so I include for completeness. However, I have trouble seeing a path to success.

In startup, George Church bets cryptocurrency will boost DNA sequencing

genomics startup co-founded by genetics pioneer George Church of Harvard emerged from stealth mode on Wednesday, proclaiming that blockchain, the technology that underlies transactions of cryptocurrencies such as bitcoin, will help people understand their genome, find cures for (unspecified) diseases, and, unlike most existing genomics companies, guarantee that individuals will retain permanent ownership of their DNA data.

Company/Drug Specific

Bristol-Myers announced positive results on an Opdivo trial; however, observers had questions due to a change in the study parameters mid-stream.

More Questions Than Answers For Bristol-Myers

Investors had been waiting like cats at a fishmonger’s for news on Bristol-Myers Squibb’s test of its two breakthrough cancer drugs, Yervoy and Opdivo, in lung cancer. This morning, Bristol said the study succeeded on a key measure. Wall Street turned up its collective nose, bidding Bristol shares down 4% as part of a larger sell-off of phama stocks.

Sanofi diabetes drug sales drop as they are excluded from two PBM’s formularies.

Want numbers on formulary pain? Sanofi’s diabetes sales are Exhibit A

As payers increasingly look to put a lid on drug costs-partly through formulary management-the decision by two leading pharmacy benefit managers to exclude Sanofi’s big-sellers in diabetes took a serious toll in 2017. Sanofi reported a U.S. diabetes sales decline of 29.5% in the fourth quarter and 22.8% for the year, to €730 million ($897 million) and €3.12 billion ($3.84 billion), respectively.

Gilead received approval for a new HIV treatment.

Gilead’s Biktarvy wins megablockbuster HIV nod, and rival GlaxoSmithKline strikes back

Gilead Sciences is all set for its next big launch. On Wednesday, a day after the company reported a disappointing hepatitis C sales outlook, the drugmaker got a needed boost with the FDA’s approval for HIV combo drug Biktarvy-a product some analysts see climbing to $10 billion.

Weekly View In-depth

Right to Try

Last week, the President mentioned the right to try bill in his State of the Union message.  While many are skeptical of the need for this bill, it continues to gain momentum. Here is a primer on the debate.

A Guide to the ‘Right to Try’ Debate

During his first State of the Union Address, U.S. President Donald Trump endorsed a proposed national “Right to Try” law, which aims to make it easier for terminally ill patients to get access to experimental drugs. The idea has widespread and bipartisan support: A bill passed unanimously in the Senate last August, and 38 states have enacted their own versions.

Patient groups do not think this bill is needed, arguing it would do more harm than good.

40 patient advocacy groups oppose ‘right to try’ drug bill

Nearly 40 patient advocacy groups told House leaders they opposed the “right to try” bill on experimental drugs, arguing the measure would “likely do more harm than good.” In a letter sent to the House leaders of both parties, the groups wrote that they believe the right to try legislation wouldn’t actually lead to greater patient access to unapproved drugs and that the current regulatory framework exists so patient protections aren’t undermined.

Concerns over the right to try bill may result in changes to the version that passed the Senate.

House eyes changes for ‘right to try’ bill

Lawmakers in the House appear likely to make changes to the “right to try” bill on experimental drugs, something supporters worry could make it harder to get the bill to President Trump Donald John Trump Tillerson: Russia already looking to interfere in 2018 midterms Dems pick up deep-red legislative seat in Missouri Speier on Trump’s desire for military parade: ‘We have a Napoleon in the making’ MORE ‘s desk.

Gottlieb believes a compromise is possible.

FDA chief optimistic on solving ‘right-to-try’ dilemma on experimental drugs 
@mhstein  |


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